(19)
(11) EP 4 323 012 A1

(12)

(43) Date of publication:
21.02.2024 Bulletin 2024/08

(21) Application number: 22788858.3

(22) Date of filing: 13.04.2022
(51) International Patent Classification (IPC): 
A61K 48/00(2006.01)
C12N 15/87(2006.01)
C07H 21/04(2006.01)
A61K 38/46(2006.01)
C12N 15/00(2006.01)
(52) Cooperative Patent Classification (CPC):
A61K 48/005; C12N 9/22; C12N 5/0621; C12N 2510/00; C12N 2506/02; C12N 2710/14043; C12N 15/1138; C12N 2310/20; C12N 15/86; A61P 27/02; A61K 38/00; C07K 14/705
(86) International application number:
PCT/US2022/024622
(87) International publication number:
WO 2022/221411 (20.10.2022 Gazette 2022/42)
(84) Designated Contracting States:
AL AT BE BG CH CY CZ DE DK EE ES FI FR GB GR HR HU IE IS IT LI LT LU LV MC MK MT NL NO PL PT RO RS SE SI SK SM TR
Designated Extension States:
BA ME
Designated Validation States:
KH MA MD TN

(30) Priority: 13.04.2021 US 202163174090 P

(71) Applicants:
  • The Trustees of Columbia University in the City of New York
    New York, NY 10027 (US)
  • University of Rochester
    Rochester, NY 14642 (US)

(72) Inventors:
  • YANG, Tingting
    New York, New York 10027 (US)
  • TSANG, Stephen, H.
    New York, New York 10027 (US)
  • ZHANG, Yu
    New York, New York 10027 (US)

(74) Representative: CH Kilger Anwaltspartnerschaft mbB 
Fasanenstraße 29
10719 Berlin
10719 Berlin (DE)

   


(54) RESCUE STRATEGIES FOR BEST1 LOSS- AND GAIN-OF-FUNCTION MUTATIONS